TACT Reviews
A non-confidential summary of all TACT reports is detailed below, however interested parties and potential funders are encouraged to contact the researcher directly to request a copy of the full TACT report. TACT does not share reports with anyone other than the original applicant but if you are interested in obtaining a copy of the report, the secretariat will try to put you in touch with the applicant. Please contact Cathy Turner for more information.
Disease :
Date : November, 2022
Meeting : Online
Applicant : Stefanie Breitenstein
Undisclosed
A small molecule with anti-inflammatory and anti-fibrotic features
A small molecule with anti-inflammatory and anti-fibrotic features
Disease :
Date : April, 2022
Meeting : Bari, Metropolitan City of Bari, Italy
Applicant : Undisclosed
Pliant Therapeutics
Development of an α7β1 Monoclonal Antibody Agonist for Treatment of Motor, Respiratory, and Cardiac Functional Decline in Patients with DMD
Plaint Therapeutics: Development of an α7β1 Monoclonal Antibody Agonist for Treatment of Motor, Respiratory, and Cardiac Functional Decline in Patients with DMD
Disease :
Date : April, 2022
Meeting : Bari, Metropolitan City of Bari, Italy
Applicant : Scott Turner
Audentes Therapeutics
AAV8-mediated exon skipping gene therapy in subjects with mutations amenable to exon skipping in the Duchenne muscular dystrophy (DMD) gene
AAV8-mediated exon skipping gene therapy in subjects with mutations amenable to exon skipping in the Duchenne muscular dystrophy (DMD) gene
Disease : Duchenne muscular dystrophy
Date : November, 2020
Meeting : Online
Applicant : Alexandra Bowden
Avidity
Phase 2 Study to Evaluate Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
Phase 2 Study to Evaluate Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
Disease : Myotonic Dystrophy
Date : September, 2020
Meeting : via teleconference
Applicant : Elizabeth Ackerman
GSK
Glaxo Smith Kline (GSK)
GSK3439171 for DMD – Non-clinical and Phase 2 Clinical Development Planning
Disease : Duchenne muscular dystrophy
Date : April, 2020
Meeting : Online Teleconference
Applicant : Victoria Ballard
NMD Pharma
Application of novel ClC-1 modulators to neuromuscular disease
Application of novel ClC-1 modulators to neuromuscular disease
Disease :
Date : April, 2020
Meeting : Online Teleconference
Applicant : John Hutchinson
Sarepta
Sarepta: Phase 3 clinical development considerations for SRP-5051 in patients with Duchenne muscular dystrophy
Sarepta: Phase 3 clinical development considerations for SRP-5051 in patients with Duchenne muscular dystrophy
Disease : Duchenne muscular dystrophy
Date : November, 2019
Meeting : Toronto, ON, Canada
Applicant : Jon Tinsley
Vertex
EX 151 Single-Cut CRISPR Gene Editing in Males with Duchenne Muscular Dystrophy (DMD)
A Study to Evaluate the Safety and Efficacy of EX 151 Single-Cut CRISPR Gene Editing in Males with Duchenne Muscular Dystrophy (DMD), with mutations amenable to exon 51 skipping or reframing
Disease : Duchenne muscular dystrophy
Date : November, 2019
Meeting : Toronto, ON, Canada
Applicant : Alison McVie Wylie
Novobioscience
Slowing and reversing heart and skeletal muscle degeneration in Duchenne muscular dystrophy (DMD) patients with the PTP1B inhibitor MSI-1436
Slowing and reversing heart and skeletal muscle degeneration in Duchenne muscular dystrophy (DMD) patients with the PTP1B inhibitor MSI-1436
Disease : Duchenne muscular dystrophy
Date : November, 2019
Meeting : Toronto, ON, Canada
Applicant : Kevin Strange
LION Therapeutics
A Single Pivotal Study of LION-101 in LGMD2I Patients
A Single Pivotal Study of LION-101 in LGMD2I Patients
Disease : Limb girdle muscular dystrophy
Date : April, 2019
Meeting : Glasgow, UK
Applicant : Sharon Hesterlee
Disease : Becker muscular dystrophy, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, Spinal muscular atrophy
Date : April, 2019
Meeting : Glasgow, UK
Applicant : Ksenija Gorni
Disease : Duchenne muscular dystrophy
Date : April, 2019
Meeting : Glasgow, UK
Applicant : Mounia Chabane
Disease : Congenital muscular dystrophy
Date : November, 2018
Meeting : Chicago, IL, USA
Applicant : Irinel Coserea
Disease : Duchenne muscular dystrophy
Date : November, 2018
Meeting : Chicago, IL, USA
Applicant : Andrea Sarasin
University of Jaén
Anti-miR106b inhibitors
Use of anti-miR106b inhibitors for promoting muscle regeneration in the treatment of Duchenne muscular dystrophy (DMD)
Disease : Duchenne muscular dystrophy
Date : November, 2018
Meeting : Chicago, IL, USA
Applicant : Amelia Aranega
Fulcrum Therapeutics
FTX1821
Reduction of DUX4 for the treatment of Facioscapulohumeral muscular dystrophy (FSHD) with FTX1821
Disease : Facioscapulohumeral muscular dystrophy
Date : July, 2018
Meeting : Vienna, Austria
Applicant : Diego Cadavid
University of the Basque Country – IIS Biodonostia
Ahulken Compounds
Ahulken Compounds: novel calcium regulators for Duchenne muscular dystrophy
Disease : Duchenne muscular dystrophy
Date : July, 2018
Meeting : Vienna, Austria
Applicant : Ainara Vallejo
University of Liverpool, UK
Corticosteroid side effects
Duchenne muscular dystrophy: Investigating corticosteroid adverse effects (DEDICAtE) study
Disease : Duchenne muscular dystrophy
Date : July, 2018
Meeting : Vienna, Austria
Applicant : Dr Hawcutt
Audentes Therapeutics
AAV8 gene therapy
Study design and endpoint selection to evaluate safety, preliminary efficacy and dose selection of AAV8 gene therapy in patients with infantile and late onset Pompe disease (IOPD and LOPD)
Disease : Pompe disease
Date : July, 2018
Meeting : Vienna, Austria
Applicant : Sal Rico
University Children’s Hospital, Basel, Switzerland
MC-DMD – Metaformin and L-Citrulline
MC-DMD - Metaformin and L-Citrulline in Duchenne Muscular Dystrophy
Disease : Duchenne muscular dystrophy
Date : October, 2017
Meeting : Montreal, QC, Canada
Applicant : Dirk Fischer
University of Western Australia
Taurine
Taurine as a therapy for Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD)
Disease : Becker muscular dystrophy, Duchenne muscular dystrophy
Date : October, 2017
Meeting : Montreal, QC, Canada
Applicant : Peter Arthur
University of Manchester, UK
Mesoangioblast-mediated exon skipping
Mesoangioblast-mediated exon skipping for genetic correction of exon 51 mutation
Disease : Duchenne muscular dystrophy
Date : October, 2017
Meeting : Montreal, QC, Canada
Applicant : Giulio Cossu
University of Basel Children's Hospital
Tamoxifen
Tamoxifen in Duchenne Muscular Dystrophy: A multicentre, randomised, double-blind, placebo controlled phase 2 safety and efficacy trial
Disease : Duchenne muscular dystrophy
Date : April, 2017
Meeting : Edinburgh, United Kingdom
Applicant : Dirk Fischer
University of Minnesota
MYOPAX
iPS cell derived myogenic progenitor cells (MYOPAX) for the treatment of muscular dystrophies
Disease : Neuromuscular diseases
Date : April, 2017
Meeting : Edinburgh, United Kingdom
Applicant : Rita Perlingeiro
Servier/ARMGO
Rycal®
Clinical development of Rycal® (Ryanodine receptor calcium release channel stabilizer) compound: S48168 (also called ARM210) in the ambulant DMD population
Disease : Duchenne muscular dystrophy
Date : April, 2017
Meeting : Edinburgh, United Kingdom
Applicant : Alexia Blesius
Disease : Myotonic Dystrophy
Date : October, 2016
Meeting : Miami, FL, USA
Applicant : Mike Snape
Disease : Becker muscular dystrophy
Date : October, 2016
Meeting : Miami, FL, USA
Applicant : Jon Tinsley
Disease : Myotubular myopathy
Date : October, 2016
Meeting : Miami, FL, USA
Applicant : Deborah Ramsdell
GlaxoSmithKline
GSK3350916A
Development of the hematopoetic prostaglandin D synthase inhibitor, GSK3350916A, for the treatment of Duchenne muscular dystrophy
Disease : Duchenne muscular dystrophy
Date : April, 2016
Meeting : Barcelona, Spain
Applicant : Patrick Eidam
Mitobridge
MTB-1
MTB-1, a selective modulator of PPARdelta(δ), for the treatment of Duchenne muscular dystrophy
Disease : Duchenne muscular dystrophy
Date : April, 2016
Meeting : Barcelona, Spain
Applicant : George Mulligan
Solid Biosciences
Simvastatin
Use of simvastatin as a potential treatment for Duchenne muscular dystrophy
Disease : Duchenne muscular dystrophy
Date : April, 2016
Meeting : Barcelona, Spain
Applicant : Jorge Quiroz
PhaseBio Pharmaceuticals Inc.
Vasomera (PB1046)
Development of PB1046 (Vasomera), as an adjunctive therapy for the treatment and prevention of cardiomyopathy associated with dystrophinopathies; Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), and X-linked dilated cardiomyopathy (XL-dCMP)
Disease : Becker muscular dystrophy, Duchenne muscular dystrophy
Date : December, 2015
Meeting : Washington, DC, United States
Applicant : Lynne Georgopolous
BioBlast Pharma Ltd.
BBRM2 (Intrathecal Azythromycin)
BBRM2 (Intrathecal Azythromycin) for the treatment of Spinal Muscular Atrophy
Disease : Spinal muscular atrophy
Date : December, 2015
Meeting : Washington, DC, United States
Applicant : Dr. Gliko-Kabir
Vall d'Hebron Research Institute (VHIR), Spain
AAV vector carrying TYMP gene
Gene Therapy for mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) using a new orphan drug consisting of an adeno-associated virus vector carrying the TYMP gene. Phase I/II clinical trial
Disease : Mitochondrial neurogastrointestinal encephalomyopathy
Date : March, 2015
Meeting : Dublin, Ireland
Applicant : Ramon Marti
Peacock Pharma, Germany
NHE-1 blocker KR-33028
The NHE-1 (sodium-hydrogen exchanger 1) blocker KR-33028 for the treatment of muscular dystrophies
Disease : Becker muscular dystrophy, Duchenne muscular dystrophy
Date : March, 2015
Meeting : Dublin, Ireland
Applicant : Stefan Schäfer
EspeRare Foundation
Rimeporide (EMD 87580)
Rimeporide (EMD 87580)- a potential disease modifying drug for Duchenne muscular dystrophy (DMD)
Disease : Duchenne muscular dystrophy
Date : March, 2015
Meeting : Dublin, Ireland
Applicant : Florence Porte-Thomé
Disease : Duchenne muscular dystrophy
Date : October, 2014
Meeting : Chicago, IL, United States
Applicant : Seth Porter, PhD
Audentes Therapeutics, Inc, San Francisco, USA
AAV8.Des.MTM1
A Phase 2 Single Arm, Open Label, Clinical Study to Evaluate the Safety and Efficacy of AAV8 Delivered Gene Therapy Delivered to Children with X-Linked Myotubular Myopathy (XLMTM)
Disease : Myotubular myopathy
Date : October, 2014
Meeting : Chicago, IL, United States
Applicant : Suyash Prasad MD
Disease : Duchenne muscular dystrophy
Date : October, 2014
Meeting : Chicago, IL, United States
Applicant : Gloria Vigliani, MD
Disease : Neuromuscular diseases
Date : May, 2014
Meeting : Berlin, Germany
Applicant : Dr Richard Franklin
University of Sheffield, UK
AAV9 viral vector
Viral vector containing DNA coding for the human SMN protein; for treatment of 5q spinal muscular atrophy
Disease : Spinal muscular atrophy
Date : May, 2014
Meeting : Berlin, Germany
Applicant : Prof Mimoun Azzouz
Disease : Duchenne muscular dystrophy
Date : November, 2013
Meeting : Newcastle upon Tyne, United Kingdom
Applicant : Rare Disease Research Unit,
Clinic for Neurology, University Medical Centre Göttingen, Göttingen, Germany
iNOS blockers
Blockade of nitric oxide-related cell stress as potential treatment for inclusion body myositis
Disease : Inclusion body myositis
Date : November, 2013
Meeting : Newcastle upon Tyne, United Kingdom
Applicant : Dr. Jens Schmidt
Disease : Duchenne muscular dystrophy
Date : November, 2013
Meeting : Newcastle upon Tyne, United Kingdom
Applicant : Dr Denis Guttridge
Disease : Duchenne muscular dystrophy
Date : April, 2013
Meeting : Baltimore, MD, United States
Applicant : Peter Flynn
Disease : Duchenne muscular dystrophy
Date : April, 2013
Meeting : Baltimore, MD, United States
Applicant : Jon Tinsley
Disease : Duchenne muscular dystrophy
Date : October, 2012
Meeting : Prague, Czech Republic
Applicant : Erica Reeves
Catabasis Pharmaceuticals, Inc
Catabasis
CAT-1004, a novel anti-inflammatory agent for treatment of Duchenne Muscular Dystrophy
Disease : Duchenne muscular dystrophy
Date : October, 2012
Meeting : Prague, Czech Republic
Applicant : Joanne M. Donovan
University of Portsmouth, UK
P2X7
P2X7 purinoceptor as a target for pharmacotherapy of Duchenne Muscular Dystrophy
Disease : Duchenne muscular dystrophy
Date : October, 2012
Meeting : Prague, Czech Republic
Applicant : Dariusz C Gorecki
Disease : Duchenne muscular dystrophy
Date : October, 2012
Meeting : Prague, Czech Republic
Applicant : Patricio Sepulveda
Italfarmaco SpA
Givinostat
A two part study to assess safety and tolerability, pharmacokinetics, effects on histology and on different clinical parameters of Givinostat in ambulant children with Duchenne Muscular Dystrophy
Disease : Duchenne muscular dystrophy
Date : April, 2012
Meeting : Arlington, VA, United States
Applicant : Paolo Bettica, MD PhD
GMP-Orphan SAS, France
Satisma
A new drug formulation development program of sodium phenylbutyrate in SMA patients
Disease : Spinal muscular atrophy
Date : April, 2012
Meeting : Arlington, VA, United States
Applicant : Fred Marin
University of Geneva, Switzerland
Tamoxifen
Proposal for clinical investigation of tamoxifen in DMD boys
Disease : Duchenne muscular dystrophy
Date : April, 2012
Meeting : Arlington, VA, United States
Applicant : Urs Ruegg & Olivier Dorchies
Tivorsan Pharmaceuticals
Biglycan
Recombinant Biglycan for Treatment of Duchenne and Becker Muscular Dystrophy
Disease : Becker muscular dystrophy, Duchenne muscular dystrophy
Date : April, 2012
Meeting : Arlington, VA, United States
Applicant : Dr. Joel Braunstein
California Stem Cell, USA
Human Embryonic Stem Cell Derived Motor Neuron Progenitors
Human Embryonic Stem Cell Derived Motor Neuron Progenitors for the Treatment of Motor Neuron Disease.
Disease : Spinal muscular atrophy
Date : October, 2011
Meeting : Lisbon, Portugal
Applicant : Chris N Airriess
Halo Therapeutics LLC, USA
HT-100 – Halofuginone
A randomised, double-blind, placebo-controlled, multiple-dose, dose-escalation study to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of HT-100 in patients with Duchenne muscular dystrophy
Disease : Duchenne muscular dystrophy
Date : October, 2011
Meeting : Lisbon, Portugal
Applicant : Marc B Blaustein MPP
NicOx SA, France
Naproxcinod – HCT 3012
A 6-month multicenter, randomised, double-blind, placebo-controlled, Phase IIa proof of principle study of naproxcinod (HCT 3012) 750 mg bid in patients with Becker Muscular Dystrophy.
Disease : Becker muscular dystrophy
Date : October, 2011
Meeting : Lisbon, Portugal
Applicant : Fabrizio Dolfi, MD
Paratek Pharmaceuticals, USA
Tetracycline Derivatives
Tetracycline Derivatives as SMN2 Splicing Modifiers for the Treatment of SMA. Screen of a tetracycline library -identified a group of potential candidates. One of these will be selected as a final candidate.
Disease : Spinal muscular atrophy
Date : January, 2011
Meeting : Charlotte, NC, United States
Applicant : Dr Higgins
Rose Pharmaceuticals, USA
GsMTx4
Therapy for Muscular Dystrophy by Inhibition of Mechanosensitive Ion Channels.
Disease : Duchenne muscular dystrophy
Date : January, 2011
Meeting : Charlotte, NC, United States
Applicant : Dr Sachs
Prothelia Inc, USA
Laminin-111
Recombinant human Laminin-111 for treatment of Duchenne Muscular Dystrophy
Disease : Duchenne muscular dystrophy
Date : June, 2010
Meeting : Barcelona, Spain
Applicant : Dr Hodges
Institute of Myology, France
N-Acetyl cysteine
Oxigem: AntiOXIdants for a GEnetic Myopathy. The First Clinical Trial for a Congenital Muscle Disorder: N-Acetyl cysteine (NAC) Treatment for SEPN1 Related Myopathy.
Disease : Congenital muscular dystrophy
Date : June, 2010
Meeting : Barcelona, Spain
Applicant : Dr Ferreiro
Phrixus Pharmaceuticals Inc, USA
P-188
An open-label, fixed dose, exploratory study to assess the efficacy and safety of P-188 NF on left ventricular volume changes in patients with Duchenne muscular dystrophy (DMD)
Disease : Duchenne muscular dystrophy
Date : June, 2010
Meeting : Barcelona, Spain
Applicant : Dr Symons
University of Messina, Messina, Italy
Flavocoxid
Randomized double-blind placebo-controlled trial of flavocoxid in Duchenne muscular dystrophy.
Disease : Duchenne muscular dystrophy
Date : February, 2010
Meeting : Rome, Italy
Applicant : Professor Giuseppe Vita and Dr. Sonia Messina
Children's National Medical Center
lisinopril / Losartan
Treatment of early cardiac systolic dysfunction in Duchenne muscular dystrophy with lisinopril or Losartan: a prospective, randomized, blinded, crossover trial.
Disease : Duchenne muscular dystrophy
Date : February, 2010
Meeting : Rome, Italy
Applicant : Dr Chris Spurney
L Sacco University Hospital, Milan, Italy
Isosorbide dinitrate plus Ibuprofen
A pharmacological treatment for muscular dystrophy combining NO-releasing and non steroidal anti-inflammatory drugs.
Disease : Duchenne muscular dystrophy
Date : February, 2010
Meeting : Rome, Italy
Applicant : Professor Clementi & Dr D'Angelo